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Molecularly driven therapies in the treatment of primary brain tumors
Ruham Alshiehk Nasany1, Macarena I de la Fuente1
1Sylvester Comprehensive Cancer Center, Division of Neuro-Oncology, Department of Neurology, University of Miami, Miami, FL, United States.
Abstract:
Molecular profiling has revolutionized the diagnosis, classification, and treatment of various cancers, with advances in next-generation sequencing and DNA methylation profiling offering unprecedented insights into tumor biology. This paradigm shift has enhanced the understanding of driver mutations in cancers translating into improved patient outcomes. In primary brain tumors, particularly gliomas, molecular profiling has redefined classification frameworks, yet meaningful improvements in patient survival remain elusive, particularly for glioblastoma. However, recent strides in molecularly targeted therapies have led to landmark FDA approvals, including agents such as vorasidenib for IDH-mutant gliomas, the combination of dabrafenib trametinib for BRAF mutated tumors, and TRK inhibitors for NTRK fusion-positive tumors. While conventional treatments like surgery, radiation, and chemotherapy remain the standard of care for gliomas, the integration of molecular-driven therapies is beginning to shape clinical management strategies. This article explores the evolving role of molecularly targeted treatments in adult primary brain tumors, examining their current applications and future potential.
Insights
Molecular profiling transforms cancer care, offering new insights into tumor biology and driving targeted therapies. While survival gains for glioblastoma are still limited, new drugs show promise for specific brain tumor mutations.
Area of Science:
- Oncology
- Genomics
- Molecular Biology
Background:
- Molecular profiling, including next-generation sequencing and DNA methylation, has advanced cancer diagnosis and classification.
- It provides deep insights into tumor biology and driver mutations, improving patient outcomes in many cancers.
- Despite advances, significant survival improvements for primary brain tumors like glioblastoma remain challenging.
Purpose of the Study:
- To explore the evolving role of molecularly targeted treatments in adult primary brain tumors.
- To examine current applications and future potential of these therapies.
- To discuss how molecular-driven therapies are shaping clinical management strategies.
Main Methods:
- Review of recent advancements in molecular profiling techniques.
- Analysis of FDA-approved molecularly targeted therapies for brain tumors.
- Examination of the integration of these therapies into standard clinical care.
Main Results:
- Molecular profiling has redefined glioma classification.
- New targeted therapies like vorasidenib, dabrafenib/trametinib, and TRK inhibitors have received FDA approval for specific mutations.
- These targeted agents are beginning to influence treatment strategies for primary brain tumors.
Conclusions:
- Molecularly targeted therapies represent a significant advancement in treating adult primary brain tumors.
- While challenges remain, particularly for glioblastoma, these therapies offer new hope and are becoming integral to personalized cancer care.
- Continued research and integration of molecular insights are crucial for improving patient survival and outcomes.
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